Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse (Q35803520)

10 August 2017

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse (English)

1 reference

10 August 2017

13

Edward H Schuchman

1 reference

10 August 2017

Marco A Passini

1

1 reference

10 August 2017

Jie Bu

2

1 reference

10 August 2017

Jonathan A Fidler

3

1 reference

10 August 2017

Robin J Ziegler

4

1 reference

10 August 2017

Joseph W Foley

5

1 reference

10 August 2017

James C Dodge

6

1 reference

10 August 2017

Wendy W Yang

7

1 reference

10 August 2017

Jennifer Clarke

8

1 reference

10 August 2017

Tatyana V Taksir

9

1 reference

10 August 2017

Denise A Griffiths

10

1 reference

10 August 2017

Michael A Zhao

11

1 reference

10 August 2017

Catherine R O'Riordan

12

1 reference

10 August 2017

Lamya S Shihabuddin

14

1 reference

10 August 2017

Seng H Cheng

15

1 reference

10 August 2017

language of work or name

English

0 references

publication date

21 May 2007

1 reference

Proceedings of the National Academy of Sciences of the United States of America

10 August 2017

1 reference

10 August 2017

104

1 reference

10 August 2017

22

1 reference

10 August 2017

9505-9510

1 reference

Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A.

10 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Gene therapy for lysosomal storage diseases.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV).

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Acid sphingomyelinase deficient mice: a model of types A and B Niemann-Pick disease

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Memory deficits associated with senescence: A neurophysiological and behavioral study in the rat

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

11 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Prevention of neuropathology in the mouse model of Hurler syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Effective gene therapy for an inherited CNS disease in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann-Pick disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Gene therapy of the brain in the dog model of Hurler's syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Effect of traumatic brain injury on mouse spatial and nonspatial learning in the Barnes circular maze.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0703509104

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1874225

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

25 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/17517638

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/17517638

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.0703509104

1 reference

10 August 2017

0 references

1 reference

10 August 2017

1 reference